Study to Evaluate the Safety and Efficacy of Daily Subcutaneous Metreleptin Treatment in Subjects With PL
- Registration Number
- NCT05164341
- Lead Sponsor
- Amryt Pharma
- Brief Summary
This is a Phase III, double-blind, placebo-controlled, safety and efficacy study of daily SC metreleptin in subjects with Partial Lipodystrophy.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 65
- Diagnosis of Familial Partial Lipodystrophy (FPLD)
- Subjects with poor metabolic control defined as:
HbA1c ≥7% (at Visit 1 and Visit 3) and/or Fasting TGs ≥500 mg/dL (5.65 mmol/L, at Visit 1 and Visit 3)
- Patients should be receiving optimized stable therapy
- Previous treatment with metreleptin
- Leptin levels >20.0 ng/mL
- Acquired or radiation induced partial lipodystrophy (APL)
Other protocol defined inclusion/exclusion criteria apply
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description metreleptin metreleptin Metreleptin \[Recombinant-methionyl human Leptin; r-metHuLeptin\] for daily injection is a sterile, white, solid lyophilised cake placebo Placebo Placebo for daily injection is a sterile, white, solid lyophilised cake
- Primary Outcome Measures
Name Time Method Percent change from Baseline to month 6 in fasting TGs in subjects with partial lipodystrophy (PL) 6 months To evaluate the efficacy (TGs) of daily SC metreleptin treatment
Change from baseline to month 6 in HbA1c in subjects with partial lipodystrophy (PL) 6 months To evaluate the efficacy (HbAa1c) of daily SC metreleptin treatment
- Secondary Outcome Measures
Name Time Method Change from baseline to months 9 and 12 in HbA1c in subjects with partial lipodystrophy (PL) 12 months To assess the effect of metreleptin on HbA1c
Percent change from baseline to months 9 and 12 in fasting TGs in subjects with partial lipodystrophy (PL) 12 months To assess the effect of metreleptin on TGs
Safety analysis of AEs, AESIs, SAEs by treatment arm 12 months To evaluate the safety of daily SC metreleptin treatment in subjects with PL
Change from Baseline to each assessment time point in quality of life (QoL) in all subjects 12 months To assess the effect of metreleptin on quality of life (QoL) in all subjects
Trial Locations
- Locations (31)
UC Davis
🇺🇸Sacramento, California, United States
Emory University
🇺🇸Atlanta, Georgia, United States
The Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Care Access Clinic
🇨🇦Sydney, Nova Scotia, Canada
University of Alabama
🇺🇸Birmingham, Alabama, United States
Flourish Boca Raton
🇺🇸Boca Raton, Florida, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
University of Missouri
🇺🇸Columbia, Missouri, United States
Amryt Research Site, Endocrinology Research Associates Inc
🇺🇸Columbus, Ohio, United States
The Ohio State University Wexner Medical Center
🇺🇸Columbus, Ohio, United States
UT Southwestern Medical Center
🇺🇸Dallas, Texas, United States
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Insight Centro de Pesquisas
🇧🇷Porto Alegre, Brazil
Hospital das Clinicas FMRP-USP
🇧🇷Ribeirão Preto, Brazil
UZ Leuven
🇧🇪Leuven, Belgium
Universitário Walter Cantídio
🇧🇷Fortaleza, Brazil
Instituto Estadual de Diabetes e Endocrinologia (IEDE-RJ)
🇧🇷Rio De Janeiro, Brazil
Nova Scotia Health
🇨🇦Halifax, Nova Scotia, Canada
Hamilton General Hospital- McMaster University
🇨🇦Hamilton, Ontario, Canada
London Health Science Centre
🇨🇦London, Ontario, Canada
Ecogene-21
🇨🇦Chicoutimi, Quebec, Canada
Hadassah University Hospital - Ein Kerem
🇮🇱Jerusalem, Israel
Leids Universitair Medisch Centrum
🇳🇱Leiden, Netherlands
Radboud University Medical Centre Interne Geneeskund
🇳🇱Nijmegen, Netherlands
Faculty of Medicine, Universite Laval
🇨🇦Québec, Quebec, Canada
Universidad Catolica
🇨🇱Santiago, Chile
Uniwersyteckie Centrum Kliniczne
🇵🇱Katowice, Poland
SPZOZ Szpital Uniwersytecki w Krakowie
🇵🇱Kraków, Poland
University Clinical Hospital of Santiago de Compostela/Hospital Universitario
🇪🇸Santiago De Compostela, Spain